The One-Stop-Shop for oligonucleotide drug development

Oligonucleotide development is a complex process, where many hurdles have to be overcome and topics have to be addressed including:

• Scientific aspects (target gene identification, oligo activity, target gen specificity, delivery)
• Pharmacological aspects (biodistribution, side effects, dose finding, toxicity, etc.)
• Preclinical regulatory aspects including Tox-study program, definition of manufacturing and analytical acceptance criteria, material planning for clinical trial, stability study, etc.

• Manufacturing aspects (e.g. method development for synthesis and purification, upscaling analytics, scalability, formulation, Fill&Filish)
• Regulatory aspects (Scientific advise, clinical program, Investigational Medicinal Product Dossier (IMPD) and other regulatory documents)

During this process, strategic decisions in early development could have dramatic impact on later clinical development, time lines and costs.

XNApharma can support your oligonucleotide development – if it is an ASO, siRNA, Aptamer, CpG or other short oligonucleotide as well as  gRNA, mRNA or a delivery approach.

We offer lab services, consulting as well as network and support for the whole process.

• ASO, siRNA, CpG, mRNA Sequence design and optimization by considering specificity, side effects, species cross-reactivity  
• Pharmacological lead optimization
• Design of ligand conjugation chemistry, e.g., GalNAc, cholesterol, peptides
• Optimization of formulation and transfection
• In vitro and in vivo efficacy analyses on mRNA (qPCR) and protein level (Western blot, ELISA, Fluorescence microscopy, etc.)

• Cytotoxicity (MTT/XTT)
• Apoptosis assays (TUNEL, cleaved PARP, etc.)
• PBMC cytokine response assay (ELISA)
• ADME, biodistribution, Dose finding
• Clinical chemistry from biological fluids
• Support regarding authority communication and Scientific Advice Meeting

Manufacturing and GMP method development are critical components of therapeutic oligonucleotide
development programs. The design and strategy of clinical studies have a significant impact on the requirements of
your GMP project, as well as on project timelines, costs, and overall development strategy. Early planning and a
clear definition of GMP requirements can help ensure an efficient and well-coordinated transition from
development to clinical application.
We provide oligonucleotide manufacturing, mRNA synthesis, LNP formulation, and Fill & Finish services to
support your early-stage therapeutic program. Our team supports your project from the
early planning stages through clinical supply, including the definition of manufacturing strategies and project
requirements. This includes the calculation of GMP material requirements, establishment of acceptance criteria,
definition of workflows and manufacturing processes, development of stability study plans, definition of validation
criteria, risk management, and other key elements of GMP project planning.
In addition, we support the preparation of regulatory documentation, including IMPDs, and assist with regulatory
interactions and communication with the relevant authorities. Our goal is to provide an integrated and efficient
approach that addresses both technical and regulatory requirements while maintaining flexibility throughout the
clinical development process.
We work closely with university hospitals, clinical research teams, and patient initiatives to
support the development and implementation of individualized clinical trials.